RARECast

548 Episodes

1. How One Drugmaker Reaches out to Communities at Greater Risk for Rare Heart Condition

   Published: 3/3/2023
2. A Next-Generation RNA Therapy Targets Telomere Disorders

   Published: 3/3/2023
3. BridgeBio Advances Therapy for Limb-Girdle Muscular Dystrophy that Started with Two Patient Families

   Published: 2/24/2023
4. Translating Urgency into Science

   Published: 2/16/2023
5. Empowering Ultra-Rare Disease Patients to Pursue the Discovery of Treatments

   Published: 2/10/2023
6. Why a Topical Cannabidiol Gel May Help Treat the Behavioral Symptoms of Fragile X

   Published: 2/2/2023
7. An Ultra-Rare Disease Drug Developer Tries to Navigate Regulatory Uncertainty

   Published: 1/26/2023
8. Powering Cells in People with Rare Mitochondrial Diseases

   Published: 1/19/2023
9. Powering Weakened and Stressed Cells in ALS to Function Better with Nanocrystal Therapy

   Published: 1/12/2023
10. Developing a New Class of Therapies Based on a Natural Cargo Carrier

    Published: 1/5/2023
11. Reaching Beyond the Limits of Enzyme Replacement Therapies with Gene Therapies

    Published: 12/29/2022
12. One Woman’s Journey as a Caregiver to a Husband with Frontotemporal Dementia

    Published: 12/22/2022
13. Treating Rare Endocrine Disorders with Therapeutic Peptides

    Published: 12/15/2022
14. Tackling the Pricing Challenges for Advanced Therapies for Rare Diseases

    Published: 12/8/2022
15. Addressing the Current Limitations of AAV Gene Therapies

    Published: 12/1/2022
16. Examining the Legislative Landscape for Rare Disease Drug Development

    Published: 11/24/2022
17. How a Drug Setback Became a Patient Community’s Gain

    Published: 11/18/2022
18. Helping Regulators and Drug Developers Understand the Challenges of Living with Fabry Disease

    Published: 11/10/2022
19. Addressing Racial Disparities in a Rare Blood Cancer

    Published: 11/3/2022
20. How New Sequencing Technology Promises to Alter the Diagnostic Odyssey

    Published: 10/27/2022